A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

Not Recruiting

Trial ID: NCT02908685


Multi-center, randomized, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of Risdiplam in adult and pediatric participants with Type 2 and Type 3 SMA. The study consists of two parts, an exploratory dose finding part (Part 1) of Risdiplam for 12 weeks and a confirmatory part (Part 2) of Risdiplam for 24 months.

Official Title

A Two Part Seamless, Multi-Center Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy Patients

Stanford Investigator(s)

John W. Day, MD, PhD
John W. Day, MD, PhD

Professor of Neurology (Adult Neurology), of Pediatrics (Genetics) and, by courtesy, of Pathology


Inclusion Criteria:

   - Confirmed diagnosis of 5q-autosomal recessive SMA

   - Negative blood pregnancy test at screening and agreement to comply with measures to
   prevent pregnancy and restrictions on sperm donation

   - For Part 1: Type 2 or 3 SMA ambulant or non-ambulant

   - For Part 2: 1) Type 2 or 3 SMA non-ambulant; 2) RULM entry item A greater than or
   equal to 2; 3) ability to sit independently as assessed by item 9 of the MFM

Exclusion Criteria:

   - Concomitant or previous participation in any investigational drug or device study
   within 90 days prior to screening, or 5 half-lives of the drug, whichever is longer

   - Concomitant or previous administration of a SMN2-targeting antisense oligonucleotide,
   SMN2 splicing modifier or gene therapy either in a clinical study or as part of
   medical care

   - Any history of cell therapy

   - Hospitalization for a pulmonary event within the last 2 months or planned at time of

   - Surgery for scoliosis or hip fixation in the one year preceding screening or planned
   within the next 18 months

   - Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular system
   diseases as considered to be clinically significant by the Investigator

   - Presence of clinically significant electrocardiogram abnormalities before study drug
   administration from average of triplicate measurement or cardiovascular disease
   indicating a safety risk for participants as determined by the Investigator

   - Any major illness within one month before the screening examination or any febrile
   illness within one week prior to screening and up to first dose administration

   - Recently initiated treatment (within less than [<] 6 months prior to randomization)
   with oral salbutamol or another beta 2-adrenergic agonist taken orally

   - Any prior use of chloroquine, hydroxychloroquine, retigabin, vigabatrin or
   thioridazine, is not allowed

   - Ascertained or presumptive hypersensitivity (e.g., anaphylactic reaction) to Risdiplam
   or to the constituents of its formulation

   - Recent history (less than one year) of ophthalmological diseases

   - Participants requiring invasive ventilation or tracheostomy


drug: Placebo

drug: Risdiplam

Not Recruiting

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305